Summary
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Developing new drugs is a costly and lengthy process. On average, it takes eight years to develop a drug, after which it can take up to three years for the drug to reach the patient. Early access is a regulated way to make drugs available more quickly by giving out-of-treatment patients access to drugs that are in the final stages of clinical development. The patients do not participate in a clinical trial, but doctors collect (real-world) data on their treatment, which the manufacturer can then use to further develop and register the drug. The purpose of mT is to (1) help out-of-treatment patients look for possible treatments, (2) support physicians in finding treatment options and realise access for patients, and (3) improve the drug development process with real-world data. Reimbursing non-registered treatments under certain conditions will not only lead to more personalised patient care, but also to an improved drug development ecosystem resulting in more affordable drugs," according to Ingmar de Gooijer.
mT operates globally and helps thousands of patients and doctors each year with information on and access to medicines in development. mT's tech platform consists of several components, including a search engine that searches clinical trial registries, medical research and compassionate use programmes (where as-yet-unapproved drugs are provided to patients under strict conditions). Pharmaceutical companies fund mT's services, which are available free of charge to doctors and patients. mT would like to develop a business case for selling early access under certain conditions in the Netherlands. In exchange for real-world data and pre- and post-authorisation price agreements, more patients will receive the care they need, the payer will have more control over the pricing of medicines and research costs for the manufacturer will be reduced. Small companies in particular benefit from this because they are often forced to collaborate with large manufacturers in the final research phase, which reduces competition. In order to establish a business case, mT set up three different pilots in which it experimented with a funding system that benefits the patient, the doctor, the payer (government or insurer) and the manufacturer.
None of the three pilots made it to the finish line. The first pilot fitted within the existing reimbursement system and could count on the support of patients' organisations and practitioners, but ran aground with the insurers. The second pilot ended when the drug manufacturer was unable to offer the therapy within the agreed terms. The third gene therapy pilot was delayed because the Ministry of Health, Welfare and Sport (VWS) was moderately enthusiastic. Ultimately, the therapy was taken over by a large pharmaceutical company, which is exactly what mT wants to avoid. So a proven business case for reimbursing early access under certain conditions does not (yet) exist in the Netherlands (although it does exist in France, where it has been used since 1994). But De Gooijer is not giving up. mT operates worldwide as a platform for information about and access to medicines in development and supports thousands of patients and doctors every year. We want to help as many patients as possible and improve the drug development model. This requires more cooperation between the parties, which we will continue to work on.'
Sometimes there is still hope for patients who have exhausted all treatment options. Medical treatments that are still under development can provide them with the necessary health benefits. myTomorrows (mT) links patients and doctors to medicines that are under development. It sounds simpler than it is.
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